Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1
Grabowski GA, Kacena K, Cole JA, Hollak CE, Zhang L, Yee J, Mistry PK, Zimran A, Charrow J, vom Dahl S. Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1. Genetics In Medicine 2009, 11: 92-100. PMID: 19265748, PMCID: PMC3793250, DOI: 10.1097/gim.0b013e31818e2c19.Peer-Reviewed Original ResearchConceptsDose-response relationshipGaucher disease type 1Type 1 patientsGaucher disease type 1 patientsDisease type 1Enzyme therapyGroup AGroup CInternational Collaborative Gaucher Group Gaucher RegistryDisease parametersType 1Propensity scoreDose-dependent improvementRoutine clinical practiceGreater treatment effectEnzyme replacement therapyNonlinear mixed-effects model analysisMixed-effects model analysisGaucher RegistryDose groupPlatelet countReplacement therapySplenic volumeIntact spleenTreatment outcomes